Exploring novel clinical trial designs for gene-based therapies proceedings of a workshop - Addie, Siobhan (National Academies Press, 2020)
Includes bibliographical references
Intro -- FrontMatter -- Reviewers -- Acknowledgments -- Contents -- Boxes, Figures, and Table -- Acronyms and Abbreviations -- 1 Introduction and Overview -- 2 Developing First-in-Human Gene Therapy Clinical Trials -- 3 Understanding the Complexities of Patient Selection, Enrollment, and the Consent Process -- 4 Developing Endpoints for Gene Therapy Clinical Trials -- 5 Integrating Gene-Based Therapies into Clinical Practice: Exploring Long-Term Clinical Follow-Up of Patients -- 6 Reflections on the Workshop and Potential Opportunities for Next Steps -- References
Appendix A: Workshop Agenda -- Appendix B: Speaker Biographical Sketches -- Appendix C: Statement of Task -- Appendix D: Registered Attendees